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ClinicalTrials.gov public records Last synced Sep 23, 2026, 6:33 AM EDT

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Showing 1–24 of 1,044 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Congenital Anemias Clear all
Active, not recruiting No phase listed Observational

Long - Term Follow Up of Sickle Cell Disease and Beta-thalassemia Subjects Previously Exposed to BIVV003 or ST-400.

NCT05145062
View directory record Official record
Conditions
Blood and Lymphatic Diseases
Interventions
BIVV003, ST-400
Drug
Lead sponsor
Sangamo Therapeutics
Industry
Eligibility
18 Years to 45 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2038
U.S. locations
7
States / cities
Oakland, California • Sacramento, California • Atlanta, Georgia + 3 more
Source: ClinicalTrials.gov public record
Updated Nov 11, 2025 · Synced Sep 23, 2026, 6:33 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Pilot Phase I/II Study of Amino Acid Leucine in Treatment of Patients With Transfusion-Dependent Diamond Blackfan Anemia

NCT01362595
View directory record Official record
Conditions
Diamond Blackfan Anemia, Blackfan Diamond Syndrome, DBA, Congenital Hypoplastic Anemia, Pure Red Cell Aplasia
Interventions
leucine
Drug
Lead sponsor
Northwell Health
Other
Eligibility
2 Years and older
Enrollment
55 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2020
U.S. locations
11
States / cities
Phoenix, Arizona • Palo Alto, California • Indianapolis, Indiana + 8 more
Source: ClinicalTrials.gov public record
Updated Dec 1, 2022 · Synced Sep 23, 2026, 6:33 AM EDT
Completed Early Phase 1 Interventional

Pilot Study of Etanercept (Enbrel) in Children With Fanconi Anemia

NCT00965666
View directory record Official record
Conditions
Fanconi Anemia
Interventions
Etanercept
Drug
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
4 Years and older
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2010
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Sep 13, 2020 · Synced Sep 23, 2026, 6:33 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Using Iodized Salt to Improve Serum Folate, B12 and Iron Levels

NCT06904612
View directory record Official record
Conditions
Neural Tube Defects, Folic Acid Deficiency, Spina Bifida, Anencephaly-Spina Bifida, B12 Deficiency Vitamin, Anemia, Iron Deficiency Anaemia Due to Dietary Causes
Interventions
Micronutrient fortified iodized salt
Dietary Supplement
Lead sponsor
Hydrocephalus and Neuroscience Institute
Other
Eligibility
18 Years to 45 Years · Female only
Enrollment
200 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022
U.S. locations
1
States / cities
Orlando, Florida
Source: ClinicalTrials.gov public record
Updated Jul 13, 2025 · Synced Sep 23, 2026, 6:33 AM EDT
Terminated No phase listed Observational

Desmopressin as a Therapy for Bedwetting in Children With Sickle Cell Disease

NCT02636387
View directory record Official record
Conditions
Nocturnal Enuresis, Anemia, Sickle Cell
Interventions
Desmopressin
Drug
Lead sponsor
Montefiore Medical Center
Other
Eligibility
8 Years to 21 Years
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
1
States / cities
The Bronx, New York
Source: ClinicalTrials.gov public record
Updated May 30, 2022 · Synced Sep 23, 2026, 6:33 AM EDT
Active, not recruiting No phase listed Observational Accepts healthy volunteers

Screening for Alpha Thalassemia in Healthy Volunteers

NCT02692872
View directory record Official record
Conditions
Alpha Thalassemia
Interventions
Not listed
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
18 Years to 39 Years
Enrollment
367 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2027
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Apr 23, 2026 · Synced Sep 23, 2026, 6:33 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-946 in Healthy Volunteers and in Participants With Sickle Cell Disease

NCT04536792
View directory record Official record
Conditions
Healthy Volunteers, Anemia, Sickle Cell
Interventions
AG-946, AG-946 Matched Placebo
Drug
Lead sponsor
Agios Pharmaceuticals, Inc.
Industry
Eligibility
18 Years to 70 Years
Enrollment
122 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2023
U.S. locations
8
States / cities
La Jolla, California • Boston, Massachusetts • New York, New York + 4 more
Source: ClinicalTrials.gov public record
Updated Apr 15, 2024 · Synced Sep 23, 2026, 6:33 AM EDT
Recruiting No phase listed Observational

Study to Understand the Genetic Risk of Developing an Immune Response After Blood Transfusions Among Individuals With Sickle Cell Disease

NCT06944067
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
2 Years to 99 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 10, 2026 · Synced Sep 23, 2026, 6:33 AM EDT
Terminated Not applicable Interventional Results available

Acupuncture for Adults With Sickle Cell Disease (SCD): A Feasibility Study

NCT04156399
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Acupuncture
Other
Lead sponsor
University of Illinois at Chicago
Other
Eligibility
18 Years and older
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Apr 11, 2022 · Synced Sep 23, 2026, 6:33 AM EDT
Completed Phase 2 Interventional

Defibrotide in Sickle Cell Disease-Related Acute Chest Syndrome

NCT03805581
View directory record Official record
Conditions
Sickle Cell Disease, Acute Chest Syndrome
Interventions
Defibrotide
Drug
Lead sponsor
New York Medical College
Other
Eligibility
2 Years to 40 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
1
States / cities
Valhalla, New York
Source: ClinicalTrials.gov public record
Updated Dec 7, 2023 · Synced Sep 23, 2026, 6:33 AM EDT
Completed Phase 1 Interventional

A Single Dose Study of the Safety, Blood Levels and Biological Effects of Aes-103 Compared to Placebo in Subjects With Stable Sickle Cell Disease

NCT01597401
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Aes-103, Placebo
Drug
Lead sponsor
Baxalta now part of Shire
Industry
Eligibility
18 Years to 65 Years
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated May 4, 2021 · Synced Sep 23, 2026, 6:33 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Adolescent, Caregiver, and Young Adult Perspectives of the Transition From Pediatric to Adult Care for Sickle Cell Disease: A Preliminary Evaluation of the Sickle Cell Disease Transition Program

NCT01569971
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Assessment
Other
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
12 Years to 30 Years
Enrollment
55 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2014
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Jun 23, 2014 · Synced Sep 23, 2026, 6:33 AM EDT
Completed Not applicable Interventional Results available

A Rehabilitation Program in Children With Sickle Cell Disease and Cognitive Deficits: a Pilot Study

NCT00895154
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
General Tutoring, Memory Training
Behavioral
Lead sponsor
Washington University School of Medicine
Other
Eligibility
8 Years to 18 Years
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2014
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated May 14, 2019 · Synced Sep 23, 2026, 6:33 AM EDT
Terminated Phase 2 Interventional Results available

SCD-Haplo: Phase II Study of HLA-Haploidentical SCT for Aggressive SCD

NCT02013375
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Haploidentical Transplant, Alemtuzumab, Total Body Irradiation, Cyclophosphamide, Sirolimus
Procedure · Drug · Radiation
Lead sponsor
Damiano Rondelli, MD
Other
Eligibility
16 Years to 60 Years
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2018
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Aug 27, 2019 · Synced Sep 23, 2026, 6:33 AM EDT
Terminated Not applicable Interventional Accepts healthy volunteers Results available

Role of Virtual Reality (VR) in Patients With Sickle Cell Disease (SCD)

NCT03387033
View directory record Official record
Conditions
Sickle Cell Disease, Virtual Reality, Anxiety, Depression, Cancer
Interventions
Relaxation response and virtual reality (VR) session
Device
Lead sponsor
Duke University
Other
Eligibility
8 Years and older
Enrollment
20 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2019
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Feb 28, 2021 · Synced Sep 23, 2026, 6:33 AM EDT
Completed Phase 2 Interventional Results available

Effect of Atorvastatin on Endothelial Dysfunction and Albuminuria in Sickle Cell Disease

NCT01732718
View directory record Official record
Conditions
Sickle Cell Disease, Sickle Cell Nephropathy
Interventions
Atorvastatin, Placebo
Drug
Lead sponsor
University of North Carolina, Chapel Hill
Other
Eligibility
18 Years to 60 Years
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2018
U.S. locations
1
States / cities
Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Mar 17, 2020 · Synced Sep 23, 2026, 6:33 AM EDT
Withdrawn Phase 2 Interventional

Efficacy and Safety Study of Multiple Doses of VIT-2763 in Adults With Transfusion-dependent Beta-thalassemia

NCT04938635
View directory record Official record
Conditions
Beta-Thalassemia
Interventions
VIT-2763 60 mg QD, VIT-2763 60 mg BID, VIT-2763 120 mg BID, Placebo
Drug
Lead sponsor
Vifor (International) Inc.
Industry
Eligibility
18 Years to 65 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
1
States / cities
Whittier, California
Source: ClinicalTrials.gov public record
Updated Nov 9, 2022 · Synced Sep 23, 2026, 6:33 AM EDT
Completed Phase 2 Interventional Results available

Hydroxyurea to Prevent Brain Injury in Sickle Cell Disease

NCT01389024
View directory record Official record
Conditions
Sickle Cell Disease, Stroke
Interventions
Hydroxyurea, Placebo
Drug
Lead sponsor
Johns Hopkins University
Other
Eligibility
12 Months to 54 Months
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2022
U.S. locations
5
States / cities
Birmingham, Alabama • Baltimore, Maryland • Kansas City, Missouri + 2 more
Source: ClinicalTrials.gov public record
Updated Jul 9, 2024 · Synced Sep 23, 2026, 6:33 AM EDT
Terminated Phase 2 Interventional

Effects of HQK-1001 in Patients With Sickle Cell Disease

NCT01601340
View directory record Official record
Conditions
Sickle Cell Disease, Sickle Cell Anemia, Sickle Cell Disorders, Hemoglobin S Disease, Sickling Disorder Due to Hemoglobin S
Interventions
HQK-1001, Placebo
Drug
Lead sponsor
HemaQuest Pharmaceuticals Inc.
Industry
Eligibility
12 Years to 60 Years
Enrollment
77 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
11
States / cities
Mobile, Alabama • Oakland, California • Washington D.C., District of Columbia + 7 more
Source: ClinicalTrials.gov public record
Updated Mar 17, 2015 · Synced Sep 23, 2026, 6:33 AM EDT
Completed Phase 2 Interventional Results available

Haploidentical Bone Marrow Transplantation in Sickle Cell Patients (BMTCTN1507)

NCT03263559
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Haploidentical Bone Marrow Transplantation, Hydroxyurea, Rabbit-ATG, Thiotepa, Fludarabine, Cyclophosphamide, Total Body Irradiation, Mesna
Procedure · Drug · Radiation
Lead sponsor
Medical College of Wisconsin
Other
Eligibility
5 Years to 45 Years
Enrollment
95 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2025
U.S. locations
32
States / cities
Birmingham, Alabama • Oakland, California • Aurora, Colorado + 24 more
Source: ClinicalTrials.gov public record
Updated Jul 7, 2026 · Synced Sep 23, 2026, 6:33 AM EDT
Completed Phase 1 Interventional

Nitric Oxide and Transfusion Therapy for Sickle Cell Patients With Pulmonary Hypertension

NCT00023296
View directory record Official record
Conditions
Sickle Cell Anemia, Pulmonary Hypertension
Interventions
Nitric Oxide, INO Pulse - NO Delivery System
Drug · Device
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
18 Years and older
Enrollment
59 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2015
U.S. locations
2
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 16, 2019 · Synced Sep 23, 2026, 6:33 AM EDT
Recruiting Not applicable Interventional

Delivering Patient-Facing Evidence-Based Guidelines Through mHealth to Adults With Sickle Cell Disease

NCT06919224
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
mHealth app + booklets
Other
Lead sponsor
Ohio State University
Other
Eligibility
18 Years and older
Enrollment
287 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2032
U.S. locations
4
States / cities
Chicago, Illinois • St Louis, Missouri • Columbus, Ohio + 1 more
Source: ClinicalTrials.gov public record
Updated Mar 22, 2026 · Synced Sep 23, 2026, 6:33 AM EDT
Terminated Phase 4 Interventional Results available

Efficacy and Safety of Ferriprox® in Patients With Sickle Cell Disease or Other Anemias

NCT02041299
View directory record Official record
Conditions
Iron Overload, Sickle Cell Disease, Other Anemias
Interventions
Deferiprone, Deferoxamine
Drug
Lead sponsor
ApoPharma
Industry
Eligibility
2 Years and older
Enrollment
230 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2019
U.S. locations
8
States / cities
Oakland, California • Chicago, Illinois • New Orleans, Louisiana + 4 more
Source: ClinicalTrials.gov public record
Updated Aug 9, 2021 · Synced Sep 23, 2026, 6:33 AM EDT
Completed Not applicable Interventional

Mindfulness-Based Intervention for Pain and Sleep in Adolescents and Young Adults With Sickle Cell Disease

NCT07402811
View directory record Official record
Conditions
Chronic Pain, Sickle Cell Disease, Sleep Disturbance
Interventions
Mobile bindfulness-based intervention
Behavioral
Lead sponsor
University of Illinois at Chicago
Other
Eligibility
15 Years to 39 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2026
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Aug 26, 2026 · Synced Sep 23, 2026, 6:33 AM EDT